Research ArticleNeurodegenerative Disease

Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

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Science Translational Medicine  25 Feb 2015:
Vol. 7, Issue 276, pp. 276ra26
DOI: 10.1126/scitranslmed.3010101

Cyclodextrin to the rescue

Niemann-Pick type C1 (NPC) disease is a severe hereditary nervous system disorder associated with the storage of cholesterol and other lipids inside nervous tissue. In new work, Vite et al. show that injection of the pharmaceutical excipient cyclodextrin into the spinal fluid of cats with naturally occurring NPC disease prevented lipids from accumulating and prevented nervous system disease from developing. The only side effect found was a loss of hearing acuity associated with therapy. This study in the cat model provides critical data on efficacy and safety of cyclodextrin administration directly into the spinal fluid that will be important for advancing this drug into clinical trials.

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